Ninerafaxstat Showcased at the 19th Annual Global Cardio Vascular Clinical Trialists (CVCT) Forum with Focus on the Potential in Non-obstructive Hypertrophic Cardiomyopathy

in Portfolio News

BOSTON, Dec. 05, 2022 (GLOBE NEWSWIRE) -- Imbria Pharmaceuticals, Inc., a clinical stage, cardiometabolic company developing novel therapies designed to enhance cellular energetics, today announced that Martin Maron, M.D., director of HCM Center at Lahey Medical Center, at the 19th Annual Global Cardio Vascular Clinical Trialists (CVCT) Forum in Washington D.C., highlighted the importance of cardiac energetic deficits in non-obstructive hypertrophic cardiomyopathy (nHCM) and how the cardiac mitotrope, ninerafaxstat, may be life changing for patients living with nHCM. Ninerafaxstat is currently being investigated in this population in the IMPROVE-HCM trial. The presentation was part of the session, “Cardiomyopathy Trials: Hypertrophic Cardiomyopathies and the Context of Rare Diseases,” that took place on December 3, 2022.

“The greatest unmet treatment need in hypertrophic cardiomyopathy (HCM) remains in patients with heart failure symptoms due to non-obstructive disease, for which there currently are no approved therapies,” said Dr. Maron. “For the majority of these HCM patients, impaired cardiac energetics is a major contributor to abnormal heart relaxation and the cause of exertional limiting symptoms. For these reasons, I am particularly enthusiastic about the opportunity that ninerafaxstat, a novel drug aimed at substantially enhancing cardiac energetics to optimize heart performance, may bring to significantly improving the quality of life for patients with HCM.”

Key presentation highlights included an overview of the unmet need in nHCM and the role of impaired cardiac energetics in the development of symptoms. In addition, Dr. Maron provided an overview of ninerafaxstat, results from the recently presented Phase 2, IMPROVE-DiCE trial (see August 26, 2022 Press Release) and the ongoing IMPROVE-HCM trial design. A copy of the presentation is available in the “Media Center” section of the Imbria website at

“There are an estimated 200,000 patients in the U.S. that suffer from nHCM,” said Jai Patel, MRCP(U.K.), chief medical officer at Imbria. “HCM is a disease of profound cardiac energy deficiency due to energy wasting by the sarcomere that results in impaired cardiac filling. Because the development of impaired energetics precedes the development of cardiac hypertrophy, modulation of cardiac energy metabolism represents a promising and unique therapeutic strategy for patients with nHCM. As our lead indication, we are evaluating ninerafaxstat in the Phase 2, IMPROVE-HCM trial to assess cardiac energetics and function, patient symptoms and maximal functional capacity (peak VO2) to directly support a Phase 3 registrational trial.”

About ninerafaxstat (formerly IMB-101)
Our lead product candidate, ninerafaxstat, is a novel, investigational cardiac mitotrope in development for a range of cardiac diseases characterized by a fundamental imbalance between energy consumption and energy supply in the heart resulting in cardiac energy deficiency. As a partial fatty acid oxidation (pFOX) inhibitor, ninerafaxstat is designed to shift cardiac substrate selection towards glucose oxidation which generates more energy in the form of ATP per unit of oxygen consumed than any other carbon substrate, increasing cardiac metabolic efficiency to support better cardiac mechanical efficiency and function. Ninerafaxstat is currently in Phase 2 clinical development in three indications: nHCM, stable angina, and heart failure with preserved ejection fraction (HFpEF).

IMPROVE-HCM is a randomized, double-blind, placebo-controlled clinical trial investigating the safety and efficacy of ninerafaxstat in patients with nHCM. This trial will measure the change from baseline of peak oxygen consumption and oxygen uptake efficiency slope, measured by standardized cardiopulmonary exercise testing in 60 nHCM patients treated with 200 mg BID of ninerafaxstat over a 12-week period. Incidence and severity of treatment emergent adverse events, as well as incidence of treatment emergent serious adverse events will also be examined. For more information, please visit (Identifier: NCT04826185).

About Imbria
Imbria is a privately held, clinical stage company developing novel therapies for patients with life-altering cardiometabolic disorders. Our clinical stage pipeline is focused on restoring or improving the cell’s ability to produce energy in cardiovascular disorders where energetic impairment is a fundamental contributor to symptoms and functional deficits. The lead product candidate, ninerafaxstat, is currently in Phase 2 clinical development in three indications: nHCM, stable angina, and HFpEF. For additional information, please visit

Komal Joshi
Imbria Pharmaceuticals, Inc.

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